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2 Objectives Since 1988, umbilical cord blood (UCB) has proven to be a viable source of stem cells for treating patients with disorders of the hematopoietic system. Collected after the routine delivery of a healthy newborn, cord blood is processed and cryopreserved in accredited laboratories following Good Manufacturing Practice (cgmp). Typically used to reconstitute the blood forming system after treatment for malignant diseases, cord blood-derived cells are being studied for potential use in novel applications such as regenerative medicine and immunotherapy. This session will: illustrate the manufacture of safe, high quality CB products describe the regulatory framework under which CBBs operate define current and potential therapeutic applications

3 Background Each year, thousands of people are diagnosed with leukemia, lymphoma, or immune system or genetic (inherited) disorders. People with these diseases have abnormal or nonfunctional blood-forming cells. A bone marrow transplant replaces a patient s diseased blood-forming cells with healthy cells. Healthy cells from another individual must be matched.

4 Stem Cell Differentiation capable of self-renewal and multi-lineage differentiation form all the cell types in a body, including placental cells give rise to all of the cell types that make up the body hematopoietic Multipotent cells can develop into more than one cell type

6 Cord Blood Is Blood that remains in the umbilical cord and placenta after a child is born Rich source of adult stem cells Similar to those found in bone marrow Obtained from a developed person Source of self-renewing production of red blood cells, platelets, and white blood cells Non-controversial Collected after baby is born No harm to mother or baby

9 Related (Family) Banking A number of private CBBs have been established worldwide Two of the largest private CBBs in US have recently reported Storing the cord blood stem cells of 700,000 newborns Each having distributed approximately 250 products for traditional transplantation and regenerative applications

10 Options for Umbilical Cord Blood Expectant parents may choose to: Donate to a public cord blood bank: Provided to patients in need of a transplant No cost Store in a private family cord blood bank: Saved for use by the donating family Associated fee Donate for research Discard as medical waste

13 Collection The delivery process is not affected by cord blood collection In Utero performed by trained OB/MW Ex Utero performed by dedicated staff Cleaning and cannulation of umbilical vein into CPD Packed, stored, and transported to the cell processing laboratory in a temperature-controlled environment

15 Processing Volume reduction and removal of red blood cells, enriched in mononuclear cell fraction Sedimentation/centrifugation Manual/automated Current Good Manufacturing Practice (cgmp) Drug rules at 21 CFR Parts 210/211 Assure safety, purity, potency, and effectiveness Standardize manufacturing processes using reagents that have been approved for human use systems and supplies that have been cleared by FDA

24 FDA Regulation & Licensure Due to intended use in allogeneic recipients, CB is classified as a biologic drug Regulated under the more stringent cgmp rules & biologics standards similar to pharmaceuticals challenges Each cord = unique biological component; cells vs. chemicals Lot sizes = single patient lot vs thousands of tablets Manufacturing process controls = drugs can be terminally sterilized; CB manufacturers rely on aseptic processing Final FDA Guidance and Draft IND Documents released Oct High profile one of the first cellular therapies to undergo process Within 2 years, all public CBBs must be licensed (20+)

30 The St. Louis Cord Blood Bank Established in 1995 Public health service collect, process, store & distribute UCB for unrelated, allogeneic transplantation Community program operate in collaboration with > 450 physicians at 29 hospitals in MO and IL, 5 hospitals in Kansas City One of the largest independent, public CBBs in the world, affiliated globally with transplant centers and registries The ultimate recycling project 2013 FDA approval to manufacture biologic drug AlloCORD

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32 2,450 SLCBB products have been distributed more than 80 different diseases in 34 countries... 20% for international patients

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34 Power of Cord Blood The chemo I had when I was eight years old killed my cancer, but along with it my bone marrow. I wasn t making anything on my own anymore. A bone marrow transplant could repopulate her blood-forming cells, but her parents and two younger sisters did not match her blood type. Before we could worry, they told us, But we have cord blood.

WMDA Policy Statement for the Utility of Autologous or Family Cord Blood Unit Storage (This policy statement has been approved and adopted by the WMDA board on the 25 th of May 2006) The Cord Blood Registries

WMDA Policy Statement on the Utility of Autologous or Family Cord Blood Unit Storage The WMDA Board adopted this policy on 25 th of May 2006. Policy updated _April 2011 The Cord Blood Working Group and

SAVE A LIFE... BY GIVING LIFE! FOLLOW US ON: HÉMA-QUÉBEC PUBLIC CORD BLOOD BANK www.hema-quebec.qc.ca Scan this code with your smart phone to access the page Register to the Public Cord Blood Bank on the

cord blood saves lives... ...by providing a rich source of blood stem cells that can be used to treat over 80 different diseases including leukemia, lymphoma, thalassemia, Tay-Sachs and sickle cell anemia.

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GAO United States Government Accountability Office Report to Congressional Committees October 2011 NATIONAL CORD BLOOD INVENTORY Practices for Increasing Availability for Transplants and Related Challenges

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1 Who is the most influential figure in cord blood around the world? What is the biggest challenge to overcome in the use of cord blood as a source of stem cells? We asked 10 leading experts in the cord

STEM CELL FACTS The ISSCR is an independent, nonproft organization providing a global forum for stem cell research and regenerative medicine. WHAT ARE STEM CELLS? Stem cells are the foundation cells for

(last updated May 13, 2004) This is an activity offered by CBBS, a CMA accredited provider. Physicians attending this course may report up to 13.25 hours of Category 1 credits toward the California Medical

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